2023
DOI: 10.3390/healthcare11101515
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Real World Evidence on the Effectiveness of Nusinersen within the National Program to Treat Spinal Muscular Atrophy in Poland

Abstract: Background: Spinal muscular atrophy (SMA) is a debilitating neuromuscular disease resulting in children’s mortality and disability. Nusinersen is available to all SMA patients in Poland since 2019. Aim: To compare mortality or disease progression to mechanical ventilation in two patient cohorts before and after the program’s introduction. Additionally, to describe the patient population treated with nusinersen and costs incurred by the public payer. Methods: We used the National Health Fund (NHF) database to i… Show more

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Cited by 4 publications
(2 citation statements)
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“…In terms of safety, nusinersen was well-tolerated within both cohorts in this study, which is consistent with recently published observational studies [ 21 , 35 , 36 ]. The occurrence of chest infections and mild restrictive lung disease is likely a feature of the disease rather than an adverse effect of nusinersen treatment.…”
Section: Discussionsupporting
confidence: 91%
“…In terms of safety, nusinersen was well-tolerated within both cohorts in this study, which is consistent with recently published observational studies [ 21 , 35 , 36 ]. The occurrence of chest infections and mild restrictive lung disease is likely a feature of the disease rather than an adverse effect of nusinersen treatment.…”
Section: Discussionsupporting
confidence: 91%
“…Furthermore, a 2021 meta-analysis that included observational data from pediatric and adult patients with SMA types II and III also reported that nusinersen provided a favorable benefit in motor performance over a 10-14-month period [35]. In terms of safety, nusinersen was well-tolerated within both cohorts in this study, which is consistent with recently published observational studies [21,36,37]. In addition, the generally improved functional performance of patients treated with nusinersen is thought to be associated with enhanced function and/or survival of motor neurons caused by increased SMN protein production.…”
Section: Discussionsupporting
confidence: 88%